11/8/26 Ultra-Rare, Ultra-Lean: Designing for a Viable ROI Under 1,000 Patients Previous Pediatric Rare Disease Development: Evidence, Flexibility, and Regulatory Pathways Next Inside the FDA Mindset - Negotiating Endpoints and Surrogates You Might Also Like OPENING KEYNOTE - From Breakthrough to Access: How Advocacy and Policy Decide What Reaches Patients. The Plausible Mechanism Pathway: A New Evidence Playbook for Rare Disease Development Who Really Decides Which Rare Disease Therapies Survive: Regulators, Investors, Payers, or Patients? Write Your Own Endpoint Justification Patient Organizations as Evidence Partners: Shaping the Science Behind Rare Disease Approval
11/8/26 Ultra-Rare, Ultra-Lean: Designing for a Viable ROI Under 1,000 Patients Previous Pediatric Rare Disease Development: Evidence, Flexibility, and Regulatory Pathways Next Inside the FDA Mindset - Negotiating Endpoints and Surrogates You Might Also Like OPENING KEYNOTE - From Breakthrough to Access: How Advocacy and Policy Decide What Reaches Patients. The Plausible Mechanism Pathway: A New Evidence Playbook for Rare Disease Development Who Really Decides Which Rare Disease Therapies Survive: Regulators, Investors, Payers, or Patients? Write Your Own Endpoint Justification Patient Organizations as Evidence Partners: Shaping the Science Behind Rare Disease Approval
